Spark Hoping Its Luxturna Will Become First FDA-Approved Gene Therapy

Last month, Spark Therapeutics submitted its gene therapy Luxturna, an experimental treatment for a rare inherited disease that causes blindness, to regulators in the United States and Europe. The company also won U.S. Food and Drug Administration designation for the experimental therapy as a treatment for a rare pediatric disease. Luxturna could be the first treatment for inherited retinal disease and the first gene therapy for a genetic disease approved in the United States. We spoke to Paulo Falabella, medical affairs ophthalmic lead for Spark Therapeutics, about Luxturna, what clinical trials to date tell us, and why this represents a significant development for not just Spark, but the entire field of gene therapy.